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High Risk Category


The overall survival for children with high risk NBL is poor and roughly 20-35%.

The disease is generally very responsive to chemotherapy, but there is a high relapse rate.

The standard treatment is generally regarded as:

Phase 1= Induction with:

Phase 2 = Consolidation with:

  • Hematopoietic stem cell rescue or transplantation (HSCT)
    • Conditioning regimen is generally carboplatin/etoposide/melphalan or busulfan/melphalan
    • TBI (low dose whole body radiation therapy) no longer used with the aim of reducing long term side effects
  • Radiation therapy to primary site after transplant to reduce risk of local recurrence

Rapid, sequential, tandem myeloablative consolidation therapy may improve the outcome

Phase 3 = Post consolidation with:

  • Immunotherapy
    • Antibodies to target GD2 present on the surface of NBL cells used i.e. chimeric anti-GD2 antibody ch14.18 combined with GM-CSF and interleukin-2 given with the retinoic acid improve event free survival
  • Retinoid
    • Oral 13-cis-retinoic acid for 6 months


COG-ANBL0532: This study is now closed to accrual.  The aim was to improve event free survival (EFS). This protocol used:

  • Dose intensification of the induction chemotherapy (with addition of dose-intensive topotecan and cyclophosphamide in induction)
    • Randomized substitution of two cycles of topotecan and cyclophosphamide for two cycles of vincristine, cyclophosphamide, and doxorubicin to assess if this improved outcome.
  • Tandem transplant using marrow support
    • Randomization between two cycles of myeloablative chemotherapy and stem cell transplantation vs a single cycle of myeloablative chemotherapy and stem cell transplantation.
  • Escalation of RT dose to improve local control


COG-ANBL0032 and COG-ANBL0931: COG non-randomized study to look at the efficacy of monoclonal antibody therapy with granulocyte-macrophage colony-stimulating factor and interleukin-2 combined with cis-retinoic acid following completion of chemotherapy


The New Approaches to Neuroblastoma Therapy (NANT) consortium is studying inclusion of myeloablative doses of iodine-131-meta-iodobenzylguanidine (MIBG) with myeloablative chemotherapy prior to stem cell transplantation in patients with an incomplete response to induction chemotherapy.


External Link:

Treatment of high risk neuroblastoma at the National Cancer Institute

Anti-GD2 Antibody with GM-CSF, Interleukin-2 and Isotretinoin for Neuroblastoma
Yu,A. et al.






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